The 2018 Orphan Drug Conference is for "Movers and Shakers" with representative from the FDA like Jacqueline Corrigan-Cury, Director, Office of Medical Policy, CDER talking on the use of RWE as well as several NIH speakers. There will be speakers on Orphan Disease programs from countries in Europe, Asia. Africa, and the Americas. Also, scientists and management from BP's such as Roche, Pfizer, Sonofi, Acceleron, Juno and Bio Martin will talk on their programs.
Drs Kaminsky and Messling will team up to discuss what is happening in Rett Syndrome. The have done this before and it is interesting that the IRS Org has chose to team with us versus Nueren. The presentation is outlined below. IMHO the P2 will be in operation before this presentation is made. All the more reason to believe that the FDA is involved with the trial design and making it more probable of success.
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Empowered educated patients influencing pre-clinical development and working with industry to optimize drug discoveryRare Disease Advocacy World
Discussion Topics
-Patient groups role guiding research, disease understanding, supporting pre-clinical development and follow up experiments
-Driving translational research and the influence on formulation, routes to delivery and overall clinical development
-Effective partnerships between advocacy and industry to expand collaborations that can support enrollment and physician engagement
Christopher MisslingChristopher Missling,President and CEO, Anavex Life Sciences
Steven KaminskySteven Kaminsky,Chief Science Officer, International Rett Syndrome Foundatio
